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临床试验/NCT01294891
NCT01294891已完成不适用

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Oregon Health and Science University1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2011年2月最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
6
试验地点
1
主要终点
Microvascular perfusion

研究概览

简要总结

The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.

研究设计

研究类型
Observational
观察模型
Case Control
时间视角
Prospective

入排标准

年龄范围
19 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • Established diagnosis of PNH or SCD Age > 18 years old

排除标准

  • Pregnant or lactating women Presence of significant right to left shunting Allergy to ultrasound contrast agent Reactive airways disease Significant peripheral or coronary artery disease

结局指标

主要结局

Microvascular perfusion

时间窗: 1 hour

1. Myocardial microvascular blood flow (MBF) and capillary blood velocity (CBV) on study day. 2. Renal MBF on study day 3. Skeletal muscle MBF and CBV on study day 4. Myocardial MBF and CBV during hyperemia 5. Skeletal muscle CBV during hyperemia

次要结局

未报告次要终点

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Jonathan R. Lindner, MD

Professor

Oregon Health and Science University

研究点 (1)

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